Recent Articles in Trials & Approvals

South Korea’s AriBio has expanded a Phase III trial of its early Alzheimer’s disease therapy into the EU, UK, Korea and China. AR1001 (mirodenafil dihydrochloride) was originally developed as an erectile dysfunction therapy targeting phosphodiesterase 5. Similar to other PDE5 therapies, it has shown efficacy for AD, though the results have been spotty for the entire PDE5 class. AriBio says AR1001 is 10-fold more potent at inhibiting PDE5 and better at entering the brain than approved PDE5 inhibitors, including sildenafil. The Phase III study is a double-blind, randomized, placebo-controlled trial with efficacy/safety endpoints after 52 weeks of treatment in early-stage AD patients. More details....

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Pittsburgh’s Viatris and Theravance of Dublin reported positive results from a China Phase III trial of their partnered maintenance therapy for COPD.  Yupelri® (revefenacin) is a once-daily nebulized long-acting muscarinic antagonist (LAMA). It works by inhibiting the M3 receptor on smooth muscle, causing bronchodilation. The Phase III placebo-controlled trial showed a statistically significant increase in trough FEV1 (forced expiratory volume in one second) versus placebo, meeting the primary efficacy endpoint. A low FEV1 suggests a breathing obstruction. Viatris has operations in Shanghai in addition to those in the US and India. More details....

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Sirius Therapeutics filed to begin a first-in-human clinical trial in Australia of SRSD107, its next-gen siRNA therapy aimed at preventing and treating thromboembolic disorders. SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA) that targets coagulation factor XI (FXI) mRNA and inhibits FXI protein expression, blocking the coagulation pathway and promoting anticoagulant/anti-thrombotic effects. In preclinical in vivo tests, a single subcutaneous dose showed near 100% reduction of FXI levels for up to six months without bleeding events. Founded in 2021, Sirius has a discovery center in San Diego and a translational medicine center in Shanghai. More details....

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Lerna Biopharma, a Singapore, Boston, Shanghai company, will present its lead drug, a first-in-class siRNA that promotes liver regeneration in patients at risk for liver failure. The candidate, LR1, takes advantage of the liver’s ability to heal itself, a long-sought goal. In preclinical testing, LR1 improved liver function and protected the liver from further degeneration. Lerna discovered the novel target using its Quartz platform, which discovers novel targets that are usually unavailable but can be addressed by siRNA technology. Lerna will describe the novel siRNA candidate at a Boston scientific convention, The Liver Meeting, later this week. More details...

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Ractigen Therapeutics, a Jiangsu RNA activation (RNAa) company, presented preclinical results of its short duplex RNA (saRNA) technology to develop a gene therapy for Duchenne muscular dystrophy (DMD). The saRNA targets activation of utrophin to increase transcription of endogenous genes, leading to restoration of their protein function. The company says saRNA offers the possibility of modulating traditionally undruggable targets, one of the few technologies that can treat diseases by stimulating the expression of therapeutic genes with insufficient expression. More details....

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